Indo-US Research Conference on Rare Diseases and Orphan Drugs

描述（由申请人提供）：估计有2500 - 3000万美国人患有罕见疾病，《孤儿药法案》将其定义为患病率为20万或更少。数以亿计的人，主要是在发展中国家，有相对常见的医疗问题，但几乎没有有效的治疗方法。这些疾病被称为被忽视的疾病，因为大多数都被政府机构和生物医学行业所忽视。一些被忽视的疾病，如疟疾和肺结核，在美国也日益成为公共卫生关注的问题。罕见病和被忽视的疾病共同造成巨大痛苦，并在医疗费用和生产力损失方面给社会造成非常高的代价。生物医学研究正在使科学家更好地了解罕见疾病和可用于开发新疗法的潜在靶点，即所谓的孤儿药和生物制剂。然而，由于药物开发失败率高和罕见病相关市场小，业界一直不愿利用这些机会。为了解决这一困境，政府和非政府组织正在寻求吸引学术科学家参与孤儿药的研究。需要科学家之间的全球合作努力，以充分利用开发罕见病新疗法的潜力。拟议的会议旨在汇集来自印度和美国的广泛的对罕见病和孤儿药感兴趣的转化和临床研究人员，分享他们的研究并促进合作。

DESCRIPTION (provided by applicant): An estimated 25-30 million Americans suffer from rare disorders, which are defined in the Orphan Drug Act as having prevalence of 200,000 or less. Hundreds of millions, primarily in developing countries, have relatively common medical problems for which there are few, if any, effective therapies. These have been dubbed neglected diseases because most have been ignored by governmental agencies and the biomedical industry. Some neglected diseases such as malaria and tuberculosis are also a growing public health concern in the United States. Together rare and neglected diseases cause enormous suffering and exact a very high cost to society in terms of medical expenses and lost productivity. Biomedical research is providing scientists with a greater understanding of rare diseases and the potential targets that can be exploited for development of new therapies, known as of orphan drugs and biologics. Industry, however, has been reluctant to capitalize on these opportunities because of the high drug development failure rate and the small markets associated with rare diseases. In an effort to address this dilemma, governmental and non-governmental organizations are seeking to attract academic scientists to orphan drug research. A global, collaborative effort among scientists is needed to fully leverage the potential to develop new therapies for rare diseases. The proposed conference is designed to bring together a wide range of translational and clinical researchers from India and the US with interests in rare diseases and orphan drugs to share their research and foster collaborations. PUBLIC HEALTH RELEVANCE: A three day conference entitled "Indo-US Conference on Rare Diseases and Orphan Drugs" will allow attendees to share their research and expertise, learn about NIH and FDA programs, and gain a better understanding of the commercialization and regulatory issues related to the orphan drug development. Conference outcomes include: 1) a better understanding of research opportunities in rare and neglected diseases, 2) establishment of new international collaborations, and 3) initiation of orphan drug discovery and development in developing countries such as India. The conference will serve as a model for future conferences intended to accelerate the worldwide development of new therapies for rare and neglected medical problems. Rare and neglected diseases are a major, worldwide public issue. Millions in the US and hundreds of millions worldwide suffer from disorders for which there are few, if any, safe and effective therapies. The Indo-US Research Conference on Rare Diseases and Orphan Drugs will bring together leading scientists from India and the US to share research, build collaborations, and increase awareness of the need for the development of orphan drugs and biologics particularly in developing countries.